barda today & tomorrow: goals, priorities, successes & … · 2015-10-14 · barda 2016...
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ASPR: Resilient People. Healthy Communities. A Nation Prepared. 0
United States Department of
Health & Human Services Office of the Assistant Secretary for Preparedness and Response
BARDA Today & Tomorrow: Goals, Priorities,
Successes & Challenges
BARDA Industry Day October 14, 2015
Dr. Robin Robinson BARDA Director
ASPR Deputy Assistant Secretary
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
Some Threats Are Very Large: Pandemic Influenza
1918: “Spanish Flu” A(H1N1)
20-100 m deaths
~500,000 in US
1957: “Asian Flu” A(H2N2)
1-4 m deaths
60-80,000 in US
1968: “Hong Kong Flu”
A(H3N 2)
1-4 m deaths
~30,000 in US 1976 Swine Flu – The “No-Show Pandemic”
2009 H1N1 Pandemic 1
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Other Threats Are Ominous: High Pathogenic Avian Influenza Viruses
H5N1
H5N1 1998 - present 2013
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Some Threats Emerge Re-emerging Infectious Diseases
H5N1 MERS-CoV
SARS-CoV 2003-2004
MERS-CoV 2012-2015
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Some Threats May Be Man-Made
• Radiological and Nuclear agents• Bacillus anthracis (anthrax)• Bacillus anthracis – Multi-drug resistant (MDR anthrax)• Botulinum toxins (botulism)• Variola virus (smallpox)• Yersinia pestis (plague)• Franciscella tularensis (tularemia)• Hemorrhagic Fever Viruses - Ebola, Marburg• Burkholderia mallei (glanders)• Burkholderia pseudomallei (melioidosis)• Rickettsia prowazekii (typhus)• Low volatility and volatile nerve agents• Hydrogen, potassium, and sodium cyanide
Pillar 1 Identify
and Assess CBRN Threats
AMERS-CoV
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Ebola is both a biothreat and an emerging infectious disease
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Threat-Based
Preparedness & Response
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Medical Countermeasures
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Medical Countermeasures
Antimicrobials Diagnostics Vaccines Medical Devices Therapeutics
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Vaccine & Drug Development is still Expensive, Risky and Lengthy
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Discovery Phase I Phase II LicensurePhase IIIPreclinicalDevelopment
Production & Delivery
PROBABILITY OF SUCCESS TO LICENSURE
PRO
DU
CT
PIPE
LIN
EPR
OD
UC
T PI
PELI
NE
LicensedProduct
1-3% 5-17% 10-25% 18-35% 45-70% 90%
IND NDA/BLA
PHASESPHASES
NIH ($11.8B)BARDA ($540M)
Project BioShield ($5.6B)
Discovery Phase I Phase II LicensurePhase IIIPreclinicalDevelopment
Production & Delivery
PROBABILITY OF SUCCESS TO LICENSURE
PRO
DU
CT
PIPE
LIN
EPR
OD
UC
T PI
PELI
NE
LicensedProduct
1-3% 5-17% 10-25% 18-35% 45-70% 90%
IND NDA/BLA
PHASESPHASES
NIH ($11.8B)BARDA ($540M)
Project BioShield ($5.6B)BARDA ARD
NIH & DoD
3-7 yr 0.5-2 yr 1-2 yr 2-3.5 yr 2.5 -4 yr 1-2 yrs TIME
$100M -130M $60-70M $70M-100M $130M-160M $190M-220M $18M-20M PIPELINE PHASE COST
Valley
of Death
Ebola MCMs 2014-2015
MERS-COV MCMs 2012-2015
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Public Health Emergency Medical Countermeasure Enterprise
(PHEMCE)
Governance Process
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Biodefense MCM History
• Project BioShield Act (2004)• Pandemic & All-Hazards Preparedness Act (2006)• Medical Countermeasure Review (2010)• Pandemic & All-Hazards Preparedness Reauthorization
Act (2013)• PHEMCE Strategic & Implementation Plan (2014)
1990-1992 1st Gulf War
Iraqi BW, CW & nuke
programs
Mid-1950’s on: DoD bio and chem defense programs
NPS founded (1999)
9/11 terrorist attacks and
anthrax letters
PHEMCE Strategy
(Mar 2007)
“Animal Rule” (May 2002)
Project BioShield (Jul 2004)
National Strategy for Pandemic Influenza
(Nov 2005)
PREP Act (Dec 2005)
PAHPA (Dec 2006) BARDA (Apr 2007)
Integrated Portfolio
(Mar 2008)
2009-H1N1 Pandemic (Apr 2009 – Aug 2010)
PHEMCE Review
(Aug 2010)
PAHPRA 2013
PHEMCE SIP
2014
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Public Health Emergency Medical Countermeasures Enterprise (PHEMCE)
Governance
ASPR/ NIH BARDA BARDA CDC/SNS CDC and CDC OPP CDC/SNS BARDA ASPR/OEM ASPR
FDFDA A equirementsR R & D Advanced
Development Acquisition
& Stockpiling Storage/
Maintenance Deployment Evaluation
Other Members:
PHEMCE COORDINATED PLANNING & EXECUTION
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: PHEMCE CBRN
Medical Countermeasure ProcessDHS
Pillar 1 Identify
and Assess Threats
BARDA
Pillar 2 Evaluate
Medical and Public Health
Consequences
ASPR/OPP
Pillar 3 Establish Medical
Countermeasure Requirements
NIH BARDA
CDC
Pillar 4 Implement
SIP for MCM Development & Acquisition
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BARDA MCM Story
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BARDA’s Mission
Support advanced development of and make available medical countermeasures for CBRN threats, pandemic influenza, and emerging infectious diseases by transitioning MCM candidates from early development across the “Valley of Death“ into advanced development to regulatory approval
O’Neil Building, Washington, DC
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BARDA Strategic Goals 2011 -2016
• Goal 1: An advanced development pipeline replete with medicalcountermeasures and platforms to address unmet public healthneeds, emphasizing innovation, flexibility, multipurpose and broadspectrum application, and long-term sustainability
• Goal 2: A capability base to provide enabling core services tomedical countermeasure innovators
• Goal 3: Agile, robust and sustainable U.S. manufacturinginfrastructure capable of rapidly producing vaccines and otherbiologics against pandemic influenza and other emerging threats
• Goal 4: Responsive and nimble programs and capabilities to addressnovel and emerging threats
• Goal 5: A ready capability to develop, manufacture and facilitatedistribution of medical countermeasures during public healthemergencies
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BARDA Key Element #1: Create Robust &Innovative MCM Development Pipeline
• ~ 200 MCM product candidates in development
0
50
100
150
200
250
2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015
EID
FLU
CBRN
PAHPA PBS MCM Review
PAHPRA H7N9 H1N1 H5N1 Ebola
MERS-CoV
BARDA ERA
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BARDA Key Element #2: Stockpile CBRN MCMs under Project BioShield
Botulism
Anthrax
Smallpox
Chemical
Radiation
Thermal Burns 2015
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BARDA Success Key #3: Seek FDA Approval
Protein Sciences Corp.
Recombinant-based Influenza Vaccine
GlaxoSmithKline
H1N1 & H5N1 Vaccines w/ Adjuvant
Anthrax Antitoxins
HGS/GSK
Botulinum Antitoxin
Cangene
Covidian
Next-Generation Portable Ventilators
Flu/RSV POC Diagnostic 3M/Focus
Novartis Cell-based Influenza Vaccine
Influenza IV Antiviral Drug
Emergent
Amgen
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BARDA Key Element #4: Establish & Nurture Partnerships
21
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BARDA Key Element #5: Establish Robust Manufacturing Capacity
• Expanding Existing Capacityby Retrofitting VaccineManufacturing Infrastructure
sanofi pasteur – Swiftwater, PA
• New Vaccine Mfg. Facilities
Novartis – Holly Springs, NC
2013 ISPE Facility of the Year
BARDA Key Element #6: Help MCM Developers for Preparedness & Response
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Fill Finish Mfg. Network
Nonclinical Development
Network
ADS Modeling
Hub
Regulatory & Quality Affairs
2009
2012
2013
2011
2014
2006
Studies Network
Clinical
CIADMs
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BARDA Key Element #7: Have the Best Technical, Clinical, & Regulatory Staff
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BARDA State of Affairs
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BARDA 2015: Harvesting ARD Bounty for PBS & Moving the Frontiers of Influenza MCMs
• Development pipeline─ Ebola Response – 12 MCMs & Natl. MCM Response Infrastructure ─ CARB launch preparations ─ More effective influenza vaccines with universal potential ─ 1st influenza immunotherapeutic development project started ─ Ebola epidemic winding down! Impacts on Vax & Rx efficacy studies
• Stockpiling─ PBS – 4 new thermal burn treatments & more MCMs on budget hold
• FDA approval─ FDA approval of Rapivab, Neupogen, AIG, Fluad, & Cobas
• Partnerships─ Partnered with FDA/CDER on Continuous Mfg. initiative - 1st project
• Manufacturing Expansion – International Flu Vax Mfg. Infrastructure• Core Services Assistance programs
─ 1st projects started in BARDA’s CIADM, FFMN, & CSN • BARDA organization – new Division leaders & Life Cycle Mgmnt.• Antigenic drift & seasonal flu vaccine mismatch HHS Action Plan
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BARDA 2016 Strategic Goals & Expectations: Expanding Horizons Amidst Uncertainties
• Development pipeline─ Formal launch CARB Action Plan for AMR Rx & Dx development ─ Launch MERS-CoV therapeutics program
• Stockpiling─ Add new & enhanced MCMs under PBS
• FDA approval─ Submit 1st IND as sponsor (H5N1 vax study) ─ Expect 2-3 FDA approved MCMs
• Partnerships─ Establish clinical study infrastructure with Saudi Arabia for MERS-CoV MCMs
• Core Services Assistance programs─ Expansion of CIADM capabilities with rapid platform technologies ─ Open BARDA’s new Innovation Hub for modeling
• BARDA organization─ Prepare new BARDA Strategic Plan 2017-2022 ─ Build permanent home for BARDA EID program
• Bioterrorism fatigue, disbelief, & denial less funding?• Global biopreparedness IOM & WHO consultations BARDA’s role
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BARDA is Always Open
BARDA Director: [email protected] 202-368-6608
BARDA - URL: http://www.phe.gov BARDA e-mail: [email protected] • Upcoming Events• PHEMCE Strategy and Implementation Plan• CBRN and Pan Flu Programs• Business Toolkit - www.phe.gov/amcg
Tech Watch program Federally-sponsored conferences Funding opportunities Resources core service programs Regulatory guidance Federal strategies and reports